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Immunotherapy

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Also Known As

Biological therapy, biotherapy, biological response modifier therapy, BRM therapy, immuno-oncology, cancer immunotherapy, immune-oncology therapy, I-O therapy, immuno-biologic therapy

Definition

Immunotherapy is a type of treatment that harnesses the body’s immune system to fight diseases, particularly cancer.1 It works by stimulating, enhancing, or suppressing the immune system’s natural ability to recognize and destroy abnormal cells.2 Unlike conventional cancer treatments that directly target cancer cells, immunotherapy focuses on empowering the immune system to better identify and attack cancer cells.3

The immune system naturally detects and destroys abnormal cells and most likely prevents or curbs the growth of many cancers.1 However, cancer cells can develop mechanisms to evade immune detection through genetic changes that make them less visible to the immune system, by expressing proteins on their surface that deactivate immune cells, or by altering the normal cells around the tumor to interfere with immune responses.1 Immunotherapy helps overcome these evasion tactics by enhancing various components of the immune response.4

Several approaches to immunotherapy have been developed, including immune checkpoint inhibitors that block proteins preventing immune cells from attacking cancer cells, T-cell transfer therapy that boosts the natural ability of T cells to fight cancer, monoclonal antibodies designed to bind to specific targets on cancer cells, treatment vaccines that boost the immune system’s response to cancer cells, and immune system modulators that enhance specific aspects of the immune response.1,5

Clinical Context

Immunotherapy is used clinically to treat various types of cancer, including but not limited to melanoma, lung cancer, bladder cancer, kidney cancer, lymphoma, and certain types of breast and colorectal cancers.1 The selection of patients for immunotherapy depends on several factors, including the type and stage of cancer, previous treatments, overall health status, and specific biomarkers such as PD-L1 expression or microsatellite instability.2,5

While immunotherapy has shown remarkable success in some patients, producing durable responses and even complete remissions, response rates vary significantly across cancer types and individual patients.2 Some patients experience dramatic and lasting responses, while others may not respond at all. This variability has led to ongoing research to identify reliable predictive biomarkers for immunotherapy response.3

The clinical implementation of immunotherapy typically involves administration through intravenous infusion for checkpoint inhibitors and most monoclonal antibodies, while some forms may be given as subcutaneous injections or oral medications.1 Treatment schedules vary depending on the specific agent, ranging from every few weeks to daily administration.4

Side effects of immunotherapy differ from those of conventional cancer treatments and are primarily related to overstimulation of the immune system, leading to autoimmune-like conditions affecting various organs including the skin, gastrointestinal tract, lungs, endocrine glands, and liver.1,4 These immune-related adverse events require prompt recognition and management, often with corticosteroids or other immunosuppressive agents.4

Beyond cancer, immunotherapy principles are also applied in treating autoimmune disorders, allergies, and infectious diseases, though the mechanisms and goals may differ significantly from cancer immunotherapy.4

Scientific Citation

[1] National Cancer Institute. Immunotherapy for Cancer. https://www.cancer.gov/about-cancer/treatment/types/immunotherapy. Updated September 24, 2019. Accessed June 2, 2025. DOI: 10.1158/2326-6066.CIR-16-0261

[2] Waldman AD, Fritz JM, Lenardo MJ. A guide to cancer immunotherapy: from T cell basic science to clinical practice. Nature Reviews Immunology. 2020;20:651-668. DOI: 10.1038/s41577-020-0306-5

[3] Kciuk M, Yahya EB, Mohamed MMI, et al. Recent Advances in Molecular Mechanisms of Cancer Immunotherapy. Cancers. 2023;15(10):2721. DOI: 10.3390/cancers15102721

[4] Vaillant AAJ, Nesseler T, Zito PM. Immunotherapy. In: StatPearls. Treasure Island (FL): StatPearls Publishing; 2023. PMID: 30020641

[5] Ribas A, Wolchok JD. Cancer immunotherapy using checkpoint blockade. Science. 2018;359(6382):1350-1355. DOI: 10.1126/science.aar4060

[6] American Cancer Society. Immunotherapy for Cancer. https://www.cancer.org/treatment/treatments-and-side-effects/treatment-types/immunotherapy.html. Updated December 27, 2022. Accessed June 2, 2025.

[7] Society for Immunotherapy of Cancer. What is Cancer Immunotherapy? https://www.sitcancer.org/clinician/cancer-immunotherapy. Accessed June 2, 2025.